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RNA-based therapeutics for Alzheimer’s disease and related tauopathies: challenges and opportunities

Year: 2026
Type: article
Abstract: Tauopathies are neurodegenerative diseases characterized by pathological tau protein accumulation. Though therapies involving monoclonal antibodies and small-molecule inhibitors have progressed, they have so far failed in multiple clinical trials, underscoring the need for innovative molecular approaches. RNA-based therapies offer an alternative disease-modifying approach by being able to target tau at its molecular origin. Diverse modalities, such as mRNA, ASO, RNAi, and SSO, offer distinct promises. Though their challenges are equally diverse, they also share common problems. This review examines the nascent field of RNA therapeutics for tauopathies, outlining emerging modalities, translational barriers, molecular targets, clinical trials, and patent trends.
Language: English
Subfield: Physiology
Field: Medicine
Open Access status: gold
Estimated APC paid: $4,060
OpenAlex ID: W7160614905
FWCI: 7.972
Cites: 99
Cited by: 2
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